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Antisense oligonucleotide therapeutic approach for Timothy syndrome

Timothy syndrome (TS) is a severe, multisystem disorder characterized by autism, epilepsy, long-QT syndrome and other neuropsychiatric conditions1. TS type 1 (TS1) is caused by a gain-of-function variant in the alternatively spliced and developmentally enriched CACNA1C exon 8A, as opposed to its counterpart exon 8. We previously uncovered several phenotypes in neurons derived from patients with TS1, including delayed channel inactivation, prolonged depolarization-induced calcium rise, impaired interneuron migration, activity-dependent dendrite retraction and an unanticipated persistent expression of exon 8A2–6. We reasoned that switching CACNA1C exon utilization from 8A to 8 would represent a potential therapeutic strategy. Here we developed antisense oligonucleotides (ASOs) to effectively decrease the inclusion of exon 8A in human cells both in vitro and, following transplantation, in vivo. We discovered that the ASO-mediated switch from exon ....

Leica Stellaris , Teledyne Lumenera , Mount Polysciences , Leica Microsystems , Takara Bio , Beckman Coulter , International Electric Company , Fisher Scientific , Stanford University , Bruker Corp , Wu Tsai Neurosciences Institute , Rapid Mr International , Thermo Fisher Scientific , Innovate Cell Technologies , Life Technologies , New England Biolabs , Tissue Kit , Gotaq Long , Supplementary Tables , One Shot Stbl , Plus Mini Kit , Strand Synthesis Supermix , Gel Doc , Worthington Biochemical , Millipore Sigma , Supplementary Table ,

QIAGEN N : Form 6-K -November 01, 2023 at 06:04 am EDT

Document

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

. ....

United States , City Of , United Kingdom , New York , Fernando Beils , Siemens Healthineers , Thomas Theuringer , Phoebe Loh , Jean Pascal Viola , John Gilardi , Daniela Berheide , Thierry Bernard , Head Of External Communications , Securities Exchange , University Mosbach School Of Business , Thermo Fisher , Danish National Genome Center , Us Securities Exchange , Life Sciences , Health Care Equipment , Research Use , Verogen Inc , Head Of Corporate Strategy Business Development , Head Of Corporate Communications , Exchange Commission , Business Administration ,

QIAGEN N.V.: QIAGEN enhances QIAwave portfolio with new eco-friendlier nucleic acid extraction kits

QIAGEN N.V.: QIAGEN enhances QIAwave portfolio with new eco-friendlier nucleic acid extraction kits
finanznachrichten.de - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from finanznachrichten.de Daily Mail and Mail on Sunday newspapers.

Baden Wüberg , Thomas Schweins , Life Sciences , Exchange Commission , Life Sciences Business Area , Research Specialist , Us Securities Exchange , University Of Pennsylvania , Frankfurt Prime Standard , Plus Mini Kit , Senior Vice President , Tissue Kit , Plasmid Miniprep Kit , Science Based Targets , Molecular Diagnostics , Exchange Act , Annual Report ,

QIAGEN enhances QIAwave portfolio with new eco-friendlier nucleic acid extraction kits

QIAGEN enhances QIAwave portfolio with new eco-friendlier nucleic acid extraction kits
tmcnet.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from tmcnet.com Daily Mail and Mail on Sunday newspapers.

Thomas Theuringer , Phoebe Loh , Daniela Berheide , Thomas Schweins , John Gilardi , Us Securities Exchange , Life Sciences Business Area , University Of Pennsylvania , Exchange Commission , Research Specialist , Life Sciences , Frankfurt Prime Standard , Plus Mini Kit , Senior Vice President , Tissue Kit , Plasmid Miniprep Kit , Science Based Targets , Molecular Diagnostics , Exchange Act , Annual Report ,

QIAGEN enhances QIAwave portfolio with new eco-friendlier nucleic acid extraction kits - Qiagen (NYSE:QGEN)

Venlo, the Netherlands, Sept. 25, 2023 (GLOBE NEWSWIRE) QIAGEN (NYSE:QGEN, Frankfurt Prime Standard: QIA)) today announced the extension of its eco-friendlier QIAwave product line with the addition of two new ....

John Gilardi , Daniela Berheide , Thomas Theuringer , Thomas Schweins , Phoebe Loh , Life Sciences , Us Securities Exchange , Research Specialist , Life Sciences Business Area , Exchange Commission , University Of Pennsylvania , Plus Mini Kit , Senior Vice President , Tissue Kit , Plasmid Miniprep Kit , Science Based Targets , Molecular Diagnostics , Exchange Act , Annual Report ,